‘Any child’s death is a tragedy’

Russian authorities launch negligence probe after 17-year-old SMA patient dies while awaiting medication

Investigators in Russia’s Novosibirsk region are looking into the circumstances surrounding the death of a 17-year-old girl, who was awaiting government-provided medication to treat spinal muscular atrophy (SMA). According to regional prosecutors, the girl was supposed to receive the medication on June 1 but died in the hospital on May 9. Investigators are still determining whether negligence on the part of government officials led to the teenager’s death. According to media reports, this is the third minor with SMA who has died in Siberia over the past year and a half. All three deaths involved patients who were waiting for the government to issue prescribed medications.

The death of a 17-year-old girl with spinal muscular atrophy (SMA) has prompted investigators in the Novosibirsk region to open a felony negligence case. According to regional investigators, preliminary data shows that the girl was in need of the drug risdiplam (marketed as Evrysdi), but wasn’t provided with it in time. The girl’s health deteriorated on May 9, and she was admitted to the hospital, where she died soon afterward.

The criminal investigation will assess whether government officials or the local administration failed to provide the girl with free medication, which resulted in the death of the minor, investigators said in a statement.

The Novosibirsk Regional Prosecutor’s Office also announced a probe into the circumstances surrounding the girl’s death. The prosecutors confirmed that the patient was 17 years old and was supposed to receive the medication on June 1.

Evgenia Goloyadova — president of the charitable foundation Save Life, which previously assisted the deceased — told the news site Tayga.Info that the girl’s death wasn’t related to a lack of the medication Evrysdi.

“The problem here isn’t that the drug wasn’t issued, the Health Ministry is in no way to blame. There is such a thing as doctors’ ignorance. And the problem is complex: [it’s] in the training of specialists, in the legislation. Any child’s death is a tragedy, especially when this death could have been prevented, but there’s no maliciousness here, you can’t assign blame.”

As Tayga.Info notes, this marks the third death of a child with SMA in Siberia over the past year and a half. In 2020, a two-year-old boy died in the Krasnoyarsk territory; he was prescribed the drug nusinersen (marketed as Spinraza), but officials didn’t provide the medication on time.

After the boy’s death, his father, Nikita Rukosuev, protested outside of the regional government building with a sign that read, “Governor, who will answer for the death of my son?”. Regional investigators opened felony cases for negligence and death by negligence. The investigation was later transferred to the Investigative Committee’s central office.

In March 2021, another boy with SMA died in the Krasnoyarsk territory just two days before his first birthday. He had been prescribed Spinraza, as well, but the drug was provided only six months later, after his condition had deteriorated. The child died in the ambulance that was supposed to bring him to the hospital to receive the medication. After his death, the territory’s Investigative Committee branch also opened a negligence case.

According to the charitable foundation SMA Families, as of November 2020, there were 1,048 patients in Russia with spinal muscular atrophy, including 823 children. The foundation estimates that there are actually 3,000–5,000 people with SMA living in Russia.

Spinraza and Evrysdi are the only two drugs registered in Russia for treating SMA. Approved for use in August 2019, Spinraza is injected into the spinal cord several times a year — a single injection costs $125,000. Evrysdi, an at-home oral treatment that has to be taken daily, was approved in November 2020 — an annual supply costs $340,000.

Despite the fact that Spinraza and Evrysdi are registered for use in Russia and are supposed to be purchased with government funds, it can be difficult for people with SMA to obtain these medications due to their high cost. As Meduza has reported previously, Russians with rare diseases often have to battle with government officials to get the medicines they need.

In other countries, there’s another drug used to treat SMA — Zolgensma. It’s priced at more than $2 million per dose, making it the most expensive medication in the world. Unlike Spinraza and Evrysdi, there are limitations on the use of Zolgensma and a patient can only receive this injection once. Zolgensma isn’t registered for use in Russia as of yet. The drug’s manufacturer applied for registration in July 2020.

The Circle of Kindness Foundation, which Russian President Vladimir Putin established in early 2021 to help children with rare diseases (using revenue from increasing personal income tax for Russia’s high-earners), has promised that all SMA patients in Russia will be provided with the medicines they need by the end of the year.

Story by Olga Korelina

Translated by Eilish Hart

Cover Photo: Kirill Kukhmar / TASS